]]>As I write this, I’m sitting in the University of British Columbia/Vancouver General Hospital (UBC/VGH) Eye Care Centre, where my husband, Chuck, is completing post-tests at the conclusion of a six-month study on prosopagnosia, otherwise known as face blindness. The condition isn’t treatable with drugs – not yet, at least – but it’s nonetheless disabling, prompting researchers at several centers in North America and Europe to work collaboratively and seek to help patients carry on with their lives.

Clinical studies of prosopagnosia have, so far, informed researchers more about the causes and structural manifestations of the disease than about potential treatments. “Acquired” face blindness can result from lesions associated with various types of brain disease or injury, such as stroke, traumatic brain injury or – as in my husband’s case – complications from brain surgery. Researchers also have discovered that some individuals are born with “congenital” or “developmental” prosopagnosia – the lifelong inability to distinguish among faces, with the possible exception of their closest family members. Many of these cases seem to run in families, prompting scientists to suspect an as-yet-undiscovered genetic component.

While not life-threatening, prosopagnosia involves visual impairments beyond the inability to recognize faces. Like many chronic illnesses, living with the condition is isolating and exhausting. Close friends and family members know they must introduce themselves every time they encounter Chuck or they’ll appear to him as a stranger. But it’s not easy to explain prosopagnosia to casual acquaintances and expect them to understand its limitations.

My husband has braved the effects of face blindness for nearly 20 years. Recently, he began treatment for depression – another outcome he has in common with many patients who turn to clinical trials seeking a fresh alternative for a chronic physical or mental illness.

All of this got me thinking about patient experiences before, during and following clinical trials and the movement to incorporate those experiences into the regulatory decision-making process.

The CFS community is not the only one feeling bruised following an adcom. In May, Aveo Oncology Inc.’s tivozanib got a 13-1 thumbs down from the FDA’s Oncologic Drugs Advisory Committee (ODAC) – and, subsequently, a CRL from the FDA – in renal cell carcinoma (RCC) based on concerns about the adequacy of the drug’s single Phase III trial, despite no overt arguments about safety. In fact, the lone ODAC vote to support the adequacy of the data came from the committee’s patient representative, who argued for approval based on the tolerability of tivozanib in RCC. A patient who testified during the ODAC public hearing said tivozanib was the first of eight drugs that had worked for him since his diagnosis four years earlier and asked committee members to keep an open mind. They didn’t.

Earlier this month, the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 9-2 against expanding the lipid-lowering agent Vascepa (icosapent ethyl) in combination with a statin drug to reduce triglycerides, non-high density lipoprotein cholesterol, apolipoprotein B, low-density lipoprotein cholesterol, total cholesterol and very low density lipoprotein cholesterol in adults. Although the drug, developed by Amarin Corp. plc, of Dublin, is already approved as monotherapy for severe hypertriglyceridemia, the committee voted to wait for results of an ongoing cardiovascular outcomes trial – not scheduled for completion until 2016 – rather than using a surrogate endpoint to recommend approval. Although the FDA has yet to issue its decision on the drug, observers worry the discussion and adcom vote could signal a deleterious shift in how the agency views efficacy requirements for chronic therapies.

Anecdotally – a word that sends shivers through FDA reviewers – the outcome of adcoms seems almost as capricious as the drugs alleged to underperform in clinical studies. Such fickleness comes at a huge cost to subjects, who join clinical trials not just in the hope of gaining pain-free days or additional months of life but also to help the larger patient community.

Chuck did not participate in the UBC/VGH clinical study with the goal of being “cured” of prosopagnosia. He volunteered so the lessons learned from his disability might help researchers develop therapies for those newly diagnosed with the disorder. Individuals who participate in drug trials do no less. Their experiences, their suggestions and their testimony before FDA adcoms deserve at least the same regard as the opinions of professionals paid to decide the fate of drugs and devices – especially when a compelling case can be made for conditional approval and post-marketing studies.

When Chuck was diagnosed with prosopagnosia 20 years ago, the first thing he asked doctors was how to treat the condition. The question was met with blank stares. Many patients suffering from disabling, disfiguring and life-threatening conditions still face similarly bleak responses. Treatments that may help even a small number of these individuals without causing greater harm than their disease deserve balanced hearings that incorporate both clinical and personal experience. Instead, adcom panels and the questions they deliberate often appear stacked for or against a particular drug, seemingly based on the preliminary judgment of FDA reviewers.

Although sponsors bear the financial costs of drug development, patients literally pay with their blood, sweat and tears. When drugs come before regulators, patients and their family members similarly deserve to be at the heart of the discussion. Certainly, clinical objectivity is necessary along the road to drug development, but so is real-world experience. Patient testimonies often inform as much or more than the experts, and their voices should not be relegated to the peanut gallery.

]]>http://www.bioworld.com/perspectives/2013/10/28/patient-experience-still-undervalued-in-regulating-drugs/feed/0Venter, Watson Warm Up for Clash of the Titanshttp://www.bioworld.com/perspectives/2012/07/10/venter-watson-warm-up-for-clash-of-the-titans/
http://www.bioworld.com/perspectives/2012/07/10/venter-watson-warm-up-for-clash-of-the-titans/#commentsTue, 10 Jul 2012 12:27:39 +0000Cormac Sheridanhttp://www.bioworld.com/perspectives/?p=1060DUBLIN ‑ Europe’s biggest scientific carnival is pitching its tent beside the River Liffey this week. Dublin is playing host to the Euroscience Open Forum (ESOF) 2012 meeting and, thereby, assumes the mantle of Europe’s City of Science for the...

]]>DUBLIN ‑ Europe’s biggest scientific carnival is pitching its tent beside the River Liffey this week. Dublin is playing host to the Euroscience Open Forum (ESOF) 2012 meeting and, thereby, assumes the mantle of Europe’s City of Science for the year.

Conceived as Europe’s answer to the annual meeting of the American Association for the Advancement of Science, ESOF has become an increasingly visible element in Europe’s efforts to foster a shared scientific identity. Although different European countries have vastly differing scientific heritages, their scientific futures are converging, thanks to an increasing level of recognition, both in Brussels and across the continent, that the region can only compete on a global basis if it acts in concert.

Ireland’s bid to host the event was originally conceived as its coming of age as a location for high quality scientific research, following an expansive strategy that began just over a decade ago. At present, however, the mood is not entirely celebratory. The present government’s commitment to open-ended, blue-skies research has wavered, following a catastrophic banking collapse in 2008, which culminated in the country entering a bailout program administered by the International Monetary Fund and the European Central Bank. Although funding for science has held up, just about, a recent sharp turn to a more industrially focused research agenda has been met with anger and dismay by some of the country’s leading scientists.

Even so, there will be plenty to celebrate in Dublin this week ‑ in terms of both homegrown successes and the very best of international science. The recent (apparent) detection of the Higgs boson at CERN, in Geneva, will provide one important focus for the meeting. CERN’s director general Rolf Heuer will be in town. But the physicists will have to share the limelight with a clutch of heavyweights from the life sciences world, including Nobel laureates Jules Hoffman (2011), Peter Doherty (1996), Torsten Niels Wiesel (1981) and James Watson (1962).

One event of interest to both camps will be Craig Venter’s What is Life lecture at Trinity College Dublin on Thursday evening. This will commemorate the original 1943 lecture series by Erwin Schroedinger, one of the founders of quantum mechanics, who spent 14 years in Dublin during and after the Second World War. Schroedinger’s book of the same name, which appeared in 1944, was a key text in the early development of molecular biology and a source of inspiration for James Watson and Francis Crick. Watson will be on hand to respond to Venter’s address. It might be an exaggeration to dub the event Clash of the Titans, but it’s an intriguing prospect nonetheless.

]]>http://www.bioworld.com/perspectives/2012/07/10/venter-watson-warm-up-for-clash-of-the-titans/feed/0The EMA’s Shambolic Handling of Glyberahttp://www.bioworld.com/perspectives/2012/05/07/the-emas-shambolic-handling-of-glybera/
http://www.bioworld.com/perspectives/2012/05/07/the-emas-shambolic-handling-of-glybera/#commentsMon, 07 May 2012 18:58:37 +0000Nuala Moranhttp://www.bioworld.com/perspectives/?p=917If you can’t see the wood for the trees the common sense response is to do a little thinning and let the light shine through. But for the bogged-down-in-bureaucracy European Medicines Agency (EMA), the response last week to the need...

]]>If you can’t see the wood for the trees the common sense response is to do a little thinning and let the light shine through.

But for the bogged-down-in-bureaucracy European Medicines Agency (EMA), the response last week to the need to increase transparency and streamline its procedures was to set up an expert committee to investigate the activities and operations of its expert committees.

I don’t imagine this is a cynical move by the recently installed head of EMA, Guido Rasi, to kick complaints about a lack of transparency and failure to listen to the needs of patients into the long grass. He knows prevarication won’t magic the issue away.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) ruled that ‑ although it had no safety concerns ‑ the small number of patients who took part in trials meant the data file did not demonstrate that Glybera is effective in limiting the acute attacks of pancreatitis that are the most insidious manifestation of the disorder.

The CHMP exercised its prerogative despite that fact that two other EMA expert bodies, the Committee for Advanced Therapies and the Scientific Advisory Group, recommended Glybera be approved under exceptional circumstances.

Rasi previously told BioWorld it was not surprising that EMA committees had reached differing conclusions, since they are independent of each other. Well maybe not, but there’s little point in preserving the independence of your experts, if the result is to render your organization sclerotic. The EMA needs to function.

What makes the case even worse is that the CHMP members who were at the meeting to consider Glybera voted 16-15 in favor of its approval. Unfortunately, it requires 17 votes for a product to be recommended for approval.

Even without knowing which way the missing 32nd member of the CHMP might have cast her or his vote, it is clear a majority of expert opinion believes Glybera should be allowed on the market and given an opportunity to build its case.

The many levels at which the shambolic handling of Glybera is reverberating are eloquently expressed in a letter that Alastair Kent, director of the UK charity The Genetic Alliance, wrote to the head of the European Commission’s Directorate for Health and Consumers protesting at the CHMP’s decision.

If there is a single good reason – beyond the procedural – why Glybera should not be approved under exceptional circumstances to allow efficacy data to accumulate, the EMA should say what it is.

]]>http://www.bioworld.com/perspectives/2012/05/07/the-emas-shambolic-handling-of-glybera/feed/0He Said What?!?! Best Biotech Quotes of 2011http://www.bioworld.com/perspectives/2011/12/29/he-said-what-best-biotech-quotes-of-2011/
http://www.bioworld.com/perspectives/2011/12/29/he-said-what-best-biotech-quotes-of-2011/#commentsThu, 29 Dec 2011 19:26:45 +0000Trista Morrisonhttp://www.bioworld.com/perspectives/?p=721As BioWorld Insight readers know, our “Word on the Street” column provides a sample of the most entertaining and thought-provoking quotes our staff stumbles upon each week. Some are gathered during interviews, some gleaned from analyst reports, and some overheard...

]]>As BioWorld Insight readers know, our “Word on the Street” column provides a sample of the most entertaining and thought-provoking quotes our staff stumbles upon each week. Some are gathered during interviews, some gleaned from analyst reports, and some overheard at conferences. As we kick off 2012, here’s a look back at some of the quotes that defined 2011:

On Business

“Biotech is all about picking the exception. Granting access to capital to everyone doesn’t strike me as the right idea.”

‑ Bob More, general partner with Frazier Healthcare Ventures, taking an optimistic view on capital constraints. Private biotechs continued to struggle in 2011, raising about as much money as in 2010.

"I think we're going to see a regression to quality."

‑ Jim Healy, general partner at Sofinnova Ventures, on how the venture contraction will mean that only the highest quality companies get funded. Several venture groups pulled out of biotech investing during 2011.

“The take-home message is that it’s easy to build a $25 million company with $100 million cash.”

‑ Tillman Gerngross, CEO of Adimab LLC, on the trend of sliding valuations post-initial public offering. Although a handful of biotechs went public in 2011, the window did not open as wide as some had hoped.

"You can't save your way to success in this business."

‑ Marina Biotech Inc.'s CEO Michael French on the tough financial choices small biotechs must make, such as accepting harsh terms to keep a company moving forward.

“Monday, I was in Chicago, presenting at ASCO. Tuesday, I’m in California, pitching at Goldman (Sachs). And Wednesday, I’m in New York, presenting at Jefferies. How many red-eye medallion miles does it take to get one deal?”

‑ A jet-lagged biopharma CEO, taking a break at the Jefferies 2011 Global Healthcare Conference, and illustrating just how hard biotechs had to work to close financings and partnerships in 2011

On Science

“What happens when the next 10 patients you see require eight different drug combinations based on the mutations in their tumors?”

‑ Outgoing ASCO president George Sledge, on the exponential increases in complexity required to tackle “chaotic” tumors with high mutational load. Personalization of medicine continued to be a theme at ASCO and AACR in 2011.

"You do have to be somewhat of a Talmudic scholar to prescribe this drug."

]]>http://www.bioworld.com/perspectives/2011/12/29/he-said-what-best-biotech-quotes-of-2011/feed/0The Good, the Bad and . . . the Huh? A 2011 Biotech Recaphttp://www.bioworld.com/perspectives/2011/12/28/the-good-the-bad-and-the-huh-a-2011-biotech-recap/
http://www.bioworld.com/perspectives/2011/12/28/the-good-the-bad-and-the-huh-a-2011-biotech-recap/#commentsWed, 28 Dec 2011 22:11:41 +0000Jennifer Boggshttp://www.bioworld.com/perspectives/?p=713The past year brought the biotech sector a mixed bag of news. Some of it was positive – for instance, the FDA picked up its drug approval pace in 2011 and, in some cases, even surprised the most hardened biotech...

The past year brought the biotech sector a mixed bag of news. Some of it was positive – for instance, the FDA picked up its drug approval pace in 2011 and, in some cases, even surprised the most hardened biotech investors by granting approval of some drugs a month or two before their PDUFA dates – while other headlines hailed discouraging trends such as the failure of Prospect Ventures to close its latest round, confirming predictions of a venture capital contraction.

As we get ready to head into 2012 (and possibly an apocalyptic count-down, if those Mayans are to be believed), let’s take a look back at some of the highs and lows of the biotech industry in 2011.

‘Me-First’ Instead of ‘Me-Too’

Biotech execs always talk about tackling unmet medical needs. So it was exciting to see in 2011 the flurry of breakthrough drugs hitting the market. An informal BioWorld poll tagged Seattle Genetics Inc.’s lymphoma drug Adcetris (brentuximab vedotin), the first approved antibody-drug conjugate, as the biggest drug approval of the year, tied with HCV game-changers Incivek (telaprevir) from Vertex Pharmaceuticals Inc. and Victrelis (boceprevir) from Merck & Co. Inc. But other notable approvals included Human Genome Sciences Inc.’s Benlysta (belimumab), the first lupus drug approved in 50-plus years, and Yervoy (ipilimumab) from Bristol-Myers Squibb Co. and Zelboraf (vemurafenib) from Roche AG and Daiichi Sankyo Co. Ltd. as the first two drugs approved for melanoma that actually improved overall survival in clinical testing. And, late in the year, Incyte Corp. scored a win with Jakafi (ruxolitinib), the first approved JAK inhibitor and the first drug for myelofibrosis.

Let’s all hope that trend of “firsts” continues in 2012.

A ‘Nobel’ Death

In a bit of irony (in an Alanis Morissette kind of way), the Nobel committee awarded one-half of the 2011 Nobel prize in physiology or medicine to Ralph Steinman, unaware that the Rockefeller University professor had died of pancreatic cancer three days earlier. Posthumous Nobels are unusual but, after brief debate, the committee wisely decided to allow Steinman to remain a Nobelist. And I’m glad. He certainly earned the title for his discovery of the dendritic cell and its role in adaptive immunity. Plus, according to BioWorld’s science editor, Anette Breindl, who heard Steinman speak in 2007, he was a pretty nice guy.

Best Bang for the Buck?

The biggest M&A deal in 2011 was easily Sanofi SA’s $20-billion-plus-contingent value rights buyout of Genzyme Corp., which followed nine months of often less-than-friendly negotiations. But it wasn’t the best deal for biotech investors. Genzyme shareholders already have lost out on the $1 CVR connected to manufacturing capacity for enzyme replacement therapies Cerezyme and Fabrazyme. Whether they will be able to earn the remaining $13 linked to multiple sclerosis drug Lemtrada (alemtuzumab) is up for debate, and, for the most part, analysts are not optimistic.

If I were to choose, I’d say that Daiichi Sankyo Co. Ltd.’s bid for Plexxikon Inc. and Shire plc’s buyout of Advanced BioHealing Inc. were far more successful M&A deals. Daiichi’s hefty $935 million payment for Plexxikon – a move validated when Zelboraf gained approval in August – came despite only getting U.S. co-promotion rights to the melanoma drug under Plexxikon’s existing partnership with Roche AG. The Shire/ABH deal, meanwhile, showcased the kind of investor returns rarely seen these days. The big pharma firm shelled out $750 million for ABH, a whopping 15x return for ABH’s largest shareholder.

‘Weighed’ Down by Safety

Despite health experts clamoring for new ways to treat the so-called obesity epidemic, drugs aimed at helping people lose weight have not had an easy time of it at the FDA. Industry observers had expected 2011 to usher in not one, but three obesity therapies; instead, all three were rejected by the FDA. Orexigen Inc.’s Contrave got stalled on cardiovascular concerns, while Vivus Inc.’s Qnexa raised worries of birth defects related to one of its generic components and Arena Pharmaceuticals Inc.’s Lorquess bumped up against carcinogenicity concerns. Contrave is set to start a large cardiovascular outcomes study, which would delay its approval until 2014. But Qnexa and Lorquess could have a shot at getting approved in 2012. That’s assuming that no additional safety issues crop up. Given the track record in the obesity space, that’s definitely no guarantee.

The Rough Side of Town

While 2011 had its much-lauded drug approvals (as noted above), the year was not without its disappointments. Two promising areas of research suffered blows – Roche pulled out of the RNAi space, dumping a high-dollar collaboration with RNAi powerhouse Alnylam Inc., while Geron Inc. abandoned its position as embryonic stem cell pioneer to focus on a less-risky cancer drug. But the biggest disappointment of the year – as chosen by 41 percent of respondents to a BioWorld poll – was the slow sales of Dendreon Corp.’s prostate cancer vaccine Provenge (sipuleucel-T). Sales fell way below estimates, and, over the course of the year, Dendreon’s shares have lost about 80 percent of their value. The Seattle-based company has since retrenched and is hoping European approval, now pending, will help accelerate revenue growth. We’ll be watching in 2012.

And the Strange Bedfellows Award Goes to . . .

Probably one of the most bizarre deals ever in biotech was 2011’s short-lived merger agreement between Allos Therapeutics Inc. and AMAG Pharmaceuticals Inc. Having in common only commercially underperforming products – Allos’ Folotyn (pralatrexate) for peripheral T-cell lymphoma and AMAG's iron deficiency drug Feraheme (ferumoxytol) – execs from both firms tried hard to convince investors and analysts of the logic in combining the companies by pointing out a handful of “cost synergies.” But the merger was met with opposition from the get-go and even prompted hedge fund MSMB Capital Management to make an unsolicited bid for AMAG. Unsurprisingly, the deal was voted down by AMAG shareholders. Seriously, what were they thinking?

Well, that’s my list of headline-grabbing biotech news in 2011. But it’s hard to remember a whole year, and I’m sure I’ve forgotten something. What do you think were the biggest events in biotech in 2011?

]]>http://www.bioworld.com/perspectives/2011/12/28/the-good-the-bad-and-the-huh-a-2011-biotech-recap/feed/0Video Coverage from #BIO2011 – State of the British Biotech Industryhttp://www.bioworld.com/perspectives/2011/06/27/state-of-the-british-biotech-industry/
http://www.bioworld.com/perspectives/2011/06/27/state-of-the-british-biotech-industry/#commentsMon, 27 Jun 2011 21:24:53 +0000Lynn Yoffeehttp://www.bioworld.com/perspectives/?p=259Nigel Gaymond, Chief Executive of the BioTech Industry, UK, discusses his opinions on the future of the British biotech sector at the 2011 BIO International Convention. Do you agree? What are you thoughts on British biotech? Let us know in...

]]>Nigel Gaymond, Chief Executive of the BioTech Industry, UK, discusses his opinions on the future of the British biotech sector at the 2011 BIO International Convention. Do you agree? What are you thoughts on British biotech? Let us know in the comments section.